Akcea Therapeutics, Inc. (NASDAQ:AKCA), an affiliate of Ionis
Pharmaceuticals, Inc. (NASDAQ:IONS) focused on developing and
commercializing drugs to treat patients with serious
cardiometabolic diseases caused by lipid disorders, today announced
the submission of a New Drug Application (NDA) to the U.S. Food and
Drug Administration (FDA) for volanesorsen, an investigational
medicine for the treatment of familial chylomicronemia syndrome
(FCS).
“The NDA submission for volanesorsen marks another important
milestone in potentially bringing volanesorsen to patients with
FCS. Patients with FCS suffer multiple, severe, daily and chronic
symptoms. We are eager to provide these patients and their
physicians with potentially the first effective tool to treat this
devastating disease,” said Paula Soteropoulos, president and chief
executive officer of Akcea. “We have successfully submitted
marketing authorization applications in the U.S., the E.U. and we
are on track to submit our application for marketing authorization
in Canada in September. We are also on track to launch volanesorsen
globally in 2018 pending approval in the respective markets.”
FCS is a severe, rare disorder characterized by extremely high
levels of triglycerides, symptoms such as abdominal pain that
affect daily living, and the risk of recurrent, potentially fatal,
acute pancreatitis. People with FCS are unable to effectively
metabolize large, triglyceride-rich lipid particles called
chylomicrons due to a deficiency in lipoprotein lipase, an enzyme
that helps to break down triglycerides. There is no effective
therapy available.
“Because of drastically impaired function of lipoprotein lipase,
patients with FCS have triglyceride levels that can reach 10 to 20
times that of healthy individuals. This frequently leads to
recurrent episodes of acute pancreatitis, which can be fatal.
Today, there is no therapy for FCS patients that can effectively
reduce their triglycerides to levels that are even close to
acceptable,” said Dr. Linda C. Hemphill of Massachusetts General
Hospital and Harvard Medical School. “I am encouraged that the
significant reductions in triglyceride levels and reduced risk of
pancreatitis in the APPROACH and COMPASS studies indicate that we
may soon have an option for these patients.”
“The entire FCS community is so thankful for Akcea and Ionis and
their dedication to FCS patients. It is because of their hard work
and determination that the community is closer to having a
treatment with the potential to vastly improve the quality of life
for those living with FCS,” said Lindsey Sutton and Melissa Goetz,
co-presidents of the FCS Foundation.
ABOUT THE VOLANESORSEN CLINICAL PROGRAM The
submission of volanesorsen for the treatment of FCS is based on
data from the Phase 3 APPROACH and COMPASS studies. The pivotal
APPROACH study, a one-year, randomized, placebo-controlled study in
66 patients with FCS (average baseline triglycerides of 2,209
mg/dL, or 25.0 mmol/L), achieved its primary endpoint of reduction
in triglycerides at three months, with a 77% mean reduction in
triglycerides, which translated into a 1,712 mg/dL (19.3 mmol/L)
mean absolute triglyceride reduction in volanesorsen-treated
patients. The treatment difference is 94% compared to an 18%
increase for placebo. In addition, in the APPROACH study, treatment
with volanesorsen was associated with a statistically significant
reduced rate of on-study pancreatitis attacks in the group of
patients who had multiple pancreatitis events during the 5 years
prior to screening and reduced abdominal pain in patients reporting
pain during the screening period.
The COMPASS study, a six-month randomized placebo-controlled
study in 113 patients with very high triglycerides (>500 mg/dL),
also achieved its primary endpoint of reduction in triglycerides at
three months, with a 71% mean reduction in triglycerides. In the
COMPASS study, treatment with volanesorsen was associated with a
statistically significant reduction in on-study pancreatitis
attacks.
The most common adverse event in the studies was injection site
reactions, which were mostly mild. Platelet count reductions were
observed in many patients. These platelet declines were not
clinically significant in most patients and were generally well
managed with monitoring and dose adjustment. Five patients
discontinued participation in the APPROACH study due to platelet
count reductions, two of which were severe; four patients
discontinued due to other nonserious adverse events.
Akcea and Ionis continue to conduct the BROADEN study, a Phase 3
clinical trial in patients with familial partial lipodystrophy
(FPL), which continues to enroll, with topline data expected in
2019. Akcea plans to file for marketing authorization for
volanesorsen to treat FPL in 2019 if the data from the BROADEN
study are positive.
The U.S. and EU regulatory agencies have granted Orphan Drug
Designation to volanesorsen for the treatment of patients with FCS.
Volanesorsen has also received Orphan Drug Designation in the EU
for the treatment of FPL.
ABOUT VOLANESORSEN, FCS AND FPL Volanesorsen, a
product of Ionis’ proprietary antisense technology, is in
development for two rare metabolic disorders: FCS and FPL.
Volanesorsen is designed to reduce the production of ApoC-III, a
protein produced in the liver that plays a central role in the
regulation of plasma triglycerides and may also affect other
metabolic parameters.
FCS is a severe, rare disorder characterized by extremely high
levels of triglycerides, daily symptoms such as abdominal pain, and
the risk of recurrent, potentially fatal, acute pancreatitis.
People with FCS are unable to effectively metabolize large,
triglyceride-rich lipid particles called chylomicrons due to a
deficiency in lipoprotein lipase, an enzyme that helps to break
down triglycerides. There is no effective therapy available.
Additional information on FCS is available at www.fcsfocus.com and
through the FCS Foundation at http://www.livingwithfcs.org and the
LPLD Alliance at www.lpldalliance.org.
FPL is a severe, rare genetic metabolic disorder characterized
by an inability of the body to store fat in normal locations. This
results in high levels of triglycerides in the bloodstream,
abnormal fat distribution around and within organs, such as the
liver and heart, and a range of metabolic abnormalities, including
severe insulin resistance. People with FPL are at increased risk of
acute pancreatitis in addition to other long-term, progressive
manifestations, such as premature cardiomyopathy, atherosclerosis,
and liver disease. Additional information on FPL is available
through Lipodystrophy United at www.lipodystrophyunited.org.
ABOUT AKCEA THERAPEUTICSAkcea Therapeutics, an
affiliate of Ionis Pharmaceuticals, Inc., is a biopharmaceutical
company focused on developing and commercializing drugs to treat
patients with serious cardiometabolic diseases caused by lipid
disorders. Akcea is advancing a mature pipeline of four novel drugs
with the potential to treat multiple diseases, including
volanesorsen, AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx and
AKCEA-APOCIII-LRx. All four drugs were discovered and are being
co-developed by Ionis, a leader in antisense therapeutics, based on
Ionis’ proprietary antisense technology. The most advanced drug in
its pipeline, volanesorsen, is under regulatory review in the U.S.
and EU for the treatment of familial chylomicronemia syndrome, or
FCS, and is currently in Phase 3 clinical development for the
treatment of familial partial lipodystrophy, or FPL. Akcea is
building the infrastructure to commercialize its drugs globally
with a focus on lipid specialists as the primary call point. Akcea
is located in Cambridge, Massachusetts. Additional information
about Akcea is available at www.akceatx.com.
FORWARD-LOOKING STATEMENTThis press release
includes forward-looking statements regarding the business of Akcea
Therapeutics, Inc. and the therapeutic and commercial potential of
volanesorsen and other products in development. Any statement
describing Akcea’s goals, expectations, financial or other
projections, intentions or beliefs is a forward-looking statement
and should be considered an at-risk statement. Such statements are
subject to certain risks and uncertainties, particularly those
inherent in the process of discovering, developing and
commercializing drugs that are safe and effective for use as human
therapeutics, and in the endeavor of building a business around
such drugs. Akcea’s forward-looking statements also involve
assumptions that, if they never materialize or prove correct, could
cause its results to differ materially from those expressed or
implied by such forward-looking statements. Although Akcea’s
forward-looking statements reflect the good faith judgment of its
management, these statements are based only on facts and factors
currently known by Akcea. As a result, you are cautioned not to
rely on these forward-looking statements. These and other risks
concerning Akcea’s programs are described in additional detail in
its final prospectus for its initial public offering and its most
recent quarterly report on Form 10-Q, which are on file with the
SEC.
In this press release, unless the context requires otherwise,
“Akcea,” “Company,” “we,” “our,” and “us” refers to Akcea
Therapeutics.
Akcea Therapeutics™ is a trademark of Ionis Pharmaceuticals,
Inc. Ionis Pharmaceuticals™ is a trademark of Ionis
Pharmaceuticals, Inc.
Media and Investor Contact:
D. Wade Walke, Ph.D.
Vice President, Corporate Communications and Investor Relations
760-603-2741
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